초록 ▼

The disclosure provides adenosine deaminases that are capable of deaminating adenosine in DNA to treat Hutchinson-Gilford progeria syndrome (HOPS). The disclosure also provides fusion proteins, guide RNAs and compositions comprising a Cas9 (., a Cas9 nickase) domain and adenosine deaminases that dea

The disclosure provides adenosine deaminases that are capable of deaminating adenosine in DNA to treat Hutchinson-Gilford progeria syndrome (HOPS). The disclosure also provides fusion proteins, guide RNAs and compositions comprising a Cas9 (., a Cas9 nickase) domain and adenosine deaminases that deaminate adenosine in DNA, for example in a gene. In some embodiments, adenosine deaminases provided herein are used to correct a C1824T mutation in. In some embodiments, the methods and compositions provided herein are used to treat Hutchinson-Gilford progeria syndrome (HGPS).

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